Going after the phenomenon of 'regRNA,' Rick Young's Camp4 secures a launch round to push two programs forward
Now, a new biotech out of Rick Young’s Whitehead Institute lab at MIT wants to introduce you to regulatory RNA, or regRNA.
The company is Camp4 Therapeutics, and it’s locked down its first fundraise with a $45 million Series A on Tuesday. Led by CEO Josh Mandel-Brehm, Camp4 wants to create oligonucleotide-based medicines that control protein expression to help regulate any kind of genetic disease.
It’s an effort that got started back in 2016, Mandel-Brehm told Endpoints News, when Young teamed up with scientific co-founder Leonard Zon at Boston Children’s Hospital. The core of their research centered on understanding how cells make decisions to control their genes, and they began mapping out cells themselves.
To do so required human cells, a “whole array” of next-generation sequencing techniques and a partner to apply a steady AI hand in generating data, Mandel-Brehm said. Young and Zon became able to control drug signaling for non-cancer indications where they could change a disease’s underlying gene expression in order to spur a therapeutic benefit.
They ran into a problem focusing originally on small molecules — the drugs worked too well, the CEO told Endpoints, altering the genes to a degree much more than they would have liked.
“They were activating too many genes. It was too nonspecific for us,” Mandel-Brehm said. “It was a good problem to have, but we asked ourselves, ‘How do we get more specific?’”
Young then reevaluated the way he thought about transcription, leading him to RNA research. While looking into the matter, he turned to a class of RNA molecules that don’t contain a core protein and are only transcribed. These RNAs had been known to researchers before, but few knew of their function because they’d get sequenced out with relative frequency.
Rather than continue ignoring these regulatory RNAs, the founders began trying to harness them for druggable purposes. The cellular maps they’d created for the earlier effort proved pivotal here, Mandel-Brehm said, because researchers can’t identify or characterize the regRNAs without them.
All of a sudden, Camp4’s platform shifted to targets. Young and Zon knew they were onto something and had one last puzzle piece to solve: how to best attack the diseases they wanted to treat, given that small molecules didn’t suit them. They settled on oligonucleotides due to the vast amount of previous research that’s gone into the field.
Over the last 15 months, Camp4 has built out its preclinical pipeline and IP, but they needed cash to take things further. They got a boost in early 2020 when they signed a licensing deal with Biogen to collaborate on microglial cells related to the CNS. But for their own pipeline, the Cambridge, MA-based biotech is aiming to initially target diseases for the liver and CNS, hoping to file INDs by the end of next year for programs targeting OTC deficiency and Dravet syndrome.
Mandel-Brehm has bigger plans than that though, as he continues charting Camp4’s path forward. The biotech has about 25 to 30 CNS disorders they’re looking at, and the CEO is aiming to be at the forefront of this new area of biology.
“The punchline is no one is able to go after upregulation diseases, but we’re taking advantage,” he said. “People have always thought of downregulation, but up is a whole different category. Our sense is it’s going to be a very hot field.”
5AM Ventures and Northpond Ventures led Tuesday’s financing alongside existing investors Andreessen Horowitz, Polaris Partners and The Kraft Group.